Updates

May 29, 2020May 29, 2020
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays ... Read more
May 27, 2020May 27, 2020
PTR-01 Shows Signs of Efficacy in RDEB Patients, Early Trial Data Reveal

Treatment with PTR-01 is well-tolerated and safe, and increases the levels of collagen type VII (C7) in the skin of adults with recessive dystrophic epidermolysis ... Read more
May 25, 2020May 25, 2020
Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 ... Read more
May 21, 2020May 21, 2020
Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, ... Read more
May 20, 2020May 20, 2020
Gentamicin Promotes Wound Closure in Severe JEB Patients, Phase 1/2 Trial Shows

When applied directly over skin wounds, the antibiotic gentamicin can restore the production of laminin 332 and promote wound closure in people with generalized ... Read more

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January 23, 2018January 23, 2018

Factors Beyond COL7A1 Mutations May Contribute to Dystrophic EB Forms, Study Suggests

A study from Spain has raised the possibility that the development of dystrophic epidermolysis bullosa (DEB) could be attributable to additional factors besides mutations in the COL7A1 gene. Researchers reached this conclusion after studying three unrelated patients with different forms of ... Read more

Updates

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

PTR-01 Shows Signs of Efficacy in RDEB Patients, Early Trial Data Reveal

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Gentamicin Promotes Wound Closure in Severe JEB Patients, Phase 1/2 Trial Shows

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Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

EMA Names CRISPR-Cas9 Gene Editing Therapy an Orphan Drug for RDEB

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Phase 3 Trial of Gene Therapy B-VEC for DEB Expected to Start by June

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Cannabinoid Cream PPP004 Receives FDA Orphan Drug Designation to Treat EB

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Oral Dapsone Can Lead to Rapid, Sustained Improvement in Inherited EB, Case Report Shows

Early Study of RDEB Gene Therapy Using SkinGun as Delivery Vehicle

Phase 1 Trial of EB Cannabinol Cream Completes Treatment in Healthy Volunteers

NORD’s Advice on COVID-19: You’re Stronger Than You Think

$75M Funding to Advance Gene Therapy for RDEB Entering Phase 3 Trial

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

Phase 1 Trial of EB Cannabinol Cream on Track Despite COVID-19, InMed Says

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

First Patient Treated in Phase 3 Trial of EB-101 Cell Therapy for RDEB

I’m Learning to Be Proud of My Body

Stress and ‘Helplessness’ Voiced by EB Caregivers in Taiwan

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Skin Care Products Used for Bathing by EB Patients Could Carry Risks

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

Factors Beyond COL7A1 Mutations May Contribute to Dystrophic EB Forms, Study Suggests

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COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

PTR-01 Shows Signs of Efficacy in RDEB Patients, Early Trial Data Reveal

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Gentamicin Promotes Wound Closure in Severe JEB Patients, Phase 1/2 Trial Shows

Subscribe to our mailing list

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

EMA Names CRISPR-Cas9 Gene Editing Therapy an Orphan Drug for RDEB

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Phase 3 Trial of Gene Therapy B-VEC for DEB Expected to Start by June

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Cannabinoid Cream PPP004 Receives FDA Orphan Drug Designation to Treat EB

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Oral Dapsone Can Lead to Rapid, Sustained Improvement in Inherited EB, Case Report Shows

Early Study of RDEB Gene Therapy Using SkinGun as Delivery Vehicle

Phase 1 Trial of EB Cannabinol Cream Completes Treatment in Healthy Volunteers

NORD’s Advice on COVID-19: You’re Stronger Than You Think

$75M Funding to Advance Gene Therapy for RDEB Entering Phase 3 Trial

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

Phase 1 Trial of EB Cannabinol Cream on Track Despite COVID-19, InMed Says

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

First Patient Treated in Phase 3 Trial of EB-101 Cell Therapy for RDEB

I’m Learning to Be Proud of My Body

Stress and ‘Helplessness’ Voiced by EB Caregivers in Taiwan

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

Skin Care Products Used for Bathing by EB Patients Could Carry Risks

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

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